Alessandra Biffi

Publications

• Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network
  Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Sep, 2019  |   Pubmed ID: 31499213
• Gene-Based Approaches to Inherited Neurometabolic Diseases
  Human Gene Therapy. 10, 2019  |   Pubmed ID: 31397176
• A Comprehensive Single Cell Transcriptional Landscape of Human Hematopoietic Progenitors
  Nature Communications. 06, 2019  |   Pubmed ID: 31160568
• Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease
  Molecular Therapy : the Journal of the American Society of Gene Therapy. 07, 2019  |   Pubmed ID: 31060789
• Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Gene Therapy Settings
  Human Gene Therapy. 09, 2019  |   Pubmed ID: 31037976
• Biodegradable Polymeric Nanoparticles Administered in the Cerebrospinal Fluid: Brain Biodistribution, Preferential Internalization in Microglia and Implications for Cell-selective Drug Release
  Biomaterials. Jul, 2019  |   Pubmed ID: 31026609
• Highly Efficient Therapeutic Gene Editing of Human Hematopoietic Stem Cells
  Nature Medicine. 05, 2019  |   Pubmed ID: 30911135
• Successful Hematopoietic Stem Cell Mobilization and Apheresis Collection Using Plerixafor Alone in Sickle Cell Patients
  Blood Advances. 10, 2018  |   Pubmed ID: 30282642
• Prostaglandin E2 Stimulates the Expansion of Regulatory Hematopoietic Stem and Progenitor Cells in Type 1 Diabetes
  Frontiers in Immunology. 2018  |   Pubmed ID: 29971065
• Metachromatic Leukodystrophy: Too Frequent (Mis)Diagnosis?
  JAMA Neurology. 08, 2018  |   Pubmed ID: 29913002
• Gene Therapy As a Curative Option for β-Thalassemia
  The New England Journal of Medicine. 04, 2018  |   Pubmed ID: 29669229
• Metallothioneins Are Neuroprotective Agents in Lysosomal Storage Disorders
  Annals of Neurology. 02, 2018  |   Pubmed ID: 29369397
• Intracerebroventricular Delivery of Hematopoietic Progenitors Results in Rapid and Robust Engraftment of Microglia-like Cells
  Science Advances. 12, 2017  |   Pubmed ID: 29226242
• Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases
  Hematology/oncology Clinics of North America. 10, 2017  |   Pubmed ID: 28895853
• Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications
  Molecular Therapy : the Journal of the American Society of Gene Therapy. 05, 2017  |   Pubmed ID: 28389320